Clinical Trial: Treatment of Rapid Onset Obesity, Hypoventilation, Hypothalamic Dysfunction, and Autonomic Dysregulation (ROHHAD )

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: High-dose Cyclophosphamide for the Treatment of ROHHAD (Rapid Onset Obesity, Hypoventilation, Hypothalamic Dysfunction, and Autonomic Dysregulation) Syndrome

Brief Summary: ROHHAD (rapid onset obesity, hypothalamic dysfunction, hypoventilation, and autonomic dysregulation) syndrome is a rare pediatric disorder associated with a cancer called neuroblastoma and presumed to be driven by an attack of the immune system on specific area in the brain. Patients develop severe symptoms and often succumb to this disease. Based on the researchers' experience the investigators conduct a clinical trial to study intensive immunosuppression with high-dose cyclophosphamide in these patients. In addition to describing the symptomatic improvement, the investigators' trial seeks to define objective markers of disease activity.

Detailed Summary:
Sponsor: Sidney Kimmel Comprehensive Cancer Center

Current Primary Outcome:

  • Symptomatic improvement on the ROHHAD Symptoms Scale of children with ROHHAD syndrome after treatment with high-dose cyclophosphamide . [ Time Frame: 18 months ]
  • Safety and toxicity of high-dose cyclophosphamide used to treat children with ROHHAD of as measured by NCI Common Terminology Criteria for Adverse Events (CTCAE) v4.0. [ Time Frame: 18 months ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • To calculate Area Under Curve (AUC) of high dose cyclophosphamide in obese children. [ Time Frame: predose, 0, 1, 2, 4, 8, 12 and 24 hrs from the completion ofinfusion. ]
  • Neuropsychological function of patients with ROHHAD before and after high-dose cyclophosphamide measured using complementary age appropriate evaluation tools including Wechsler Preschool and Primary Scale of Intelligence RAN objects, and AIM. [ Time Frame: 18 months ]


Original Secondary Outcome: Same as current

Information By: Sidney Kimmel Comprehensive Cancer Center

Dates:
Date Received: April 8, 2015
Date Started: March 2015
Date Completion: March 2023
Last Updated: May 17, 2016
Last Verified: May 2016